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CIRM Directors Pleased with Performance Audit Findings

By Dr. Matthew Watson


The $3 billion California stem cell
agency received a "very favorable" performance audit report
compared to other government agencies, CIRM directors were told
today.

Representatives of Moss Adams, which
was paid $234,944 by CIRM for the study, made the comments during a
presentation today to the agency's 29 directors. During their
comments, CIRM executives and directors focused on the favorable
aspects of the findings of the six-month study.
CIRM Chairman J.T. Thomas said the
report showed that CIRM is "doing better than being on the right
track." Co-vice chairman Art Torres said,

 "Comparatively we
have done very well."

The report praised the professionalism
of the CIRM staff – "a high caliber group" – and noted
the seven-year-old agency is both "ramping up and ramping down"
at the same time – a reference to the end of state bond funding for
CIRM in 2017.
Prior to the presentation, CIRM
President Alan Trounson said the staff would review the findings and
come up with a plan for the board at its July meeting. The agency is
already implementing some of the recommendations.
The audit was required by a recent
state law that also allowed CIRM to hire more than 50 persons, a cap
imposed by Proposition 71, which created the agency. The audit found a need for improvement in 27 areas and made recommendations. Of the 20 recommendations with the highest priority,
half involved how CIRM manages its information, much of which is
needed for good decision-making. The audit did not assess the
scientific performance of the agency.
The Moss Adams report, performed by the
Seattle firm's San Francisco office, said,

"CIRM board members and senior
management do not receive regularly updated, enterprise-level
performance information. The ability to evaluate performance against
strategic goals is critical to effective leadership and program
monitoring, evaluation, and reporting. CIRM does not currently have a
formal performance reporting program."

In addition to decision-making
information, Moss Adams called for improvements in the agency's
long-troubled grants management system, better grant outcome
tracking, development of a results-based communications plan,
creation of a comprehensive, formal business development plan,
formulation of a comprehensive information technology plan that would
include steps to establish clear responsibility for CIRM's website
and improved monitoring of invention disclosure forms from grantee
institutions.
Last week, in a long overdue move, the
agency hired a director for information technology, who is expected
to solve many of the problems cited in the audit.
State law requires another performance audit in a few years. 

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Live Coverage of Tomorrow’s California Stem Cell Meeting

By Dr. Matthew Watson


The California Stem Cell Report will
provide live coverage of tomorrow's meeting of the governing board of
the $3 billion California stem cell agency. Directors are expected to
make major decisions about the agency's future direction, hear the results
of the first-ever performance audit and award about $95 million in
grants or loans.

The meeting will be held near the San
Francisco airport with another public teleconference location at UC
San Francisco
. Los Angeles will also have two public teleconference
locations. Another will be in La Jolla.
The meeting will be audiocast live on
the Internet, which the California Stem Cell Report will monitor from its
base in Panama near the Pacific entrance to the Panama Canal.
Instructions for listening in on the
audiocast can be found on the agenda along with specific addresses
for the public teleconference locations. The meeting is scheduled to
begin at 9 a.m. PDT.  

Source:
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Michael J. Fox Backs Away From Stem Cell Cure for Parkinson’s

By Dr. Matthew Watson


It was not exactly a case of "Back
to the Future,
" the hit movie starring Michael J. Fox,
but it did offer a reflection on the past.

It involves the actor's
changing views on stem cell research in connection with Parkinson's
disease, which he has had since the 1990s.
Fox's 2004 Ad
Early on, Fox was well-known for his
support of human embryonic stem cell research. ABC News
recently described him as having become "one of the country’s
most visible advocates for stem cell research." In California, Fox was a prominent promoter of the ballot initiative, Proposition 71,
that created the $3 billion California stem cell agency in 2004. "
He filmed a TV commercial that was
aired widely during the 2004 campaign to create the stem cell agency,
declaring,

 "It could save the life of someone you love."

Today he is considerably less
confident. In an interview last week with ABC, he cited "problems
along the way." Fox said,

“It’s not so much that [stem cell
research has] diminished in its prospects for breakthroughs as much
as it’s the other avenues of research have grown and multiplied and
become as much or more promising. So, an answer may come from stem
cell research but it’s more than likely to come from another area.”

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Third Researcher Appealing Grant Rejection to Stem Cell Agency Board

By Dr. Matthew Watson


The director of robotics and biosurgery
at UC Davis is appealing rejection of his application for a
$4.9 million grant from the California stem cell agency.

The scientist, W. Douglas Boyd,
noted that his proposal was given a scientific score of 67, which was
one point below the cutoff for most grants approved by CIRM's
Grants Working Group
. Thirteen grants fell in the 68 to 53
range, including Boyd's. Reviewers approved four in that range,
including two with scores of 53.
Boyd's letter was brief, focusing on a
letter of support from an Indiana firm, Cook Biotech, Inc.,
that would supply the "material and technical expertise to
create a new bioengineered cardiac patch
material."
The appeal letter, along with other
appeals(see here and here), will be given to CIRM directors in the agenda material for their meeting tomorrow in San Francisco. The
board does not have to act on the petitions or discuss them.
Researchers can also appear before the board to make a case.

Claire Pomeroy, CEO of the UC
Davis Health
Systems, is a member of the CIRM board. She
will be barred from taking part in any discussion of Boyd's
application or voting on it.

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Stem Cell Agency Hires Tech Chief to Solve a Myriad of Problems

By Dr. Matthew Watson


In a move that was long overdue, the $3
billion California stem cell agency last week hired a director of
information technology to straighten out key problems ranging from
its grants management system to how it handles its website.

The new hire comes as the CIRM
governing board faces the results of its first-ever performance audit, which is markedly critical of how the agency handles its
information. Half of the audit's 20 highest priority recommendations for improvement focus on information deficiencies, including
critical information necessary for CIRM executives to determine the
agency's performance.
Solving those problems will fall on
the shoulders of Bill Gimbel, who is no stranger to CIRM. He has been
working with the agency as an information technology advisor since
2010 through a contract with Infonetica, Inc., of Pleasanton, Ca., according to CIRM spokesman Kevin McCormack.  Gimbel is now the first staff person in a chief technology position at CIRM since October 2007, when about 25 percent of CIRM employees left.
Bill Gimbel
A graduate of MIT, Gimbel, who will be
paid $180,000 annually, has a broad range of experience in computer
technology and software dating back to 1992. According to his
Linkedin web site, he was most recently director of IT at Infonetica.
He lists himself as owner of aptReader, an app for reference books.
He has also worked for LearningExpress and Scholastic, Inc.
The stem cell agency has been wrestling with information technology issues for years. The critical grants
management system has been an issue at least since 2007, when
directors were told its costs would not exceed $757,000. No figures
for the total spent since then have been made public by CIRM, which
is attempting to build a custom system, but the amount clearly and
easily surpasses the 2007 estimate, based on some of the outside
consulting costs. Although the agency hopes to resolve many of the
problems by the end of this calendar year, the grant system was the
target of considerable attention by Moss Adams, the firm that
prepared the performance audit.
Over the years, CIRM directors have received
intermittent, sketchy CIRM staff reports about the grants management
system, but the Moss Adams discussion is the most
comprehensive.
Among other things, the Moss Adams report said in bureaucratically delicate language,

 "Integration
of website content management has not been an integral part of the
GMS (grants management system) development process, which could
result in suboptimal operational efficiency and effectiveness.

"Grants management system
development is effectively managed at a tactical level, but it lacks
dedicated, strategic governance and oversight, which has resulted in
an elongated development process and requirements conflicts."

Moss Adams said,

"The new grants management system
intellectual property module, currently under development, does not
include provisions to address commercialization activity."

The performance audit additionally
said,

"CIRM board members and senior
management do not receive regularly updated, enterprise-level
performance information. The ability to evaluate performance against
strategic goals is critical to effective leadership and program
monitoring, evaluation, and reporting. CIRM does not currently have a
formal performance reporting program."

Moss Adams continued on other
information technology topics:

"Data and document access are
inefficient as a result of CIRM operating without a document
management system.....In most cases, CIRM staff cannot access
information without human interface. Information is stored in
multiple locations, which are not linked or indexed."

The audit said that the agency has
tried to solve its information problems without a plan. 

 "CIRM’s
information system needs have been met by a variety of tools,
including in-house developed applications, off-the-shelf
applications, databases, and spreadsheets, most of which are not
integrated," the audit stated.

One of the effects of all this is much
wasted time when CIRM's tiny staff tries to extract information from
the hodge-podge of systems. It is time that cannot be spared as the
workload increases in the next few years, as it is certain to do. 
CIRM's 29-member board is scheduled to consider the performance audit at its meeting this Thursday. 

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SD scientists get $18 million in stem cell funds

By daniellenierenberg

San Diego scientists will receive about $18.1 million in the latest round of funding from the California Institute of Regenerative Medicine (CIRM), the agency that's distributing $3 billion in stem cell research money made available through Proposition 71.

Since funding began, San Diego County researchers have been awarded at least $258 million, making the region one of the largest stem cell research clusters in the country.

Extended U-T science coverage on Facebook

Here's a sample of the latest grants:

Mark Tuszynski, UC San Diego, $4.7 million for research on novel stem cell therapies to treat spinal cord injuries.

Peter Schutlz, The Scripps Research Institute, $4.3 million for research to treat multiple sclerosis.

Juan Carlos Izpisua Belmonte, Salk Institute, $2.3 million for research that would help repair damaged blood vessels.

Yang Xu, UC San Diego, $1.8 million for research that would help treat heart failure.

Eric Adler, UC San Diego, $1.7 million for research to help treat Danon disease, which causes major abnormalities in heart and skeletal muscles.

David Schubert, Salk Institute, $1.7 million for research aimed at treating Alzheimer's disease

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Former Zumba instructor with cancer encourages Hispanics to donate bone marrow

By Dr. Matthew Watson

Photo by Rachel Denny Clow, Corpus Christi Caller-Times // Buy this photo

Rachel Denny Clow/Caller-Times Cristina Rodriguez sits with her dogs Coby (left) and Flower at her home Thursday. Rodriguez, who has non-Hodgkin lymphoma, is having a Zumba benefit on Sunday and inviting people to register to donate bone marrow. Rodriguez is a former Zumba instructor.

CORPUS CHRISTI Had Cristina Rodriguez's cancer been more aggressive, had it penetrated her bones, things might have been different.

And while she has had chemotherapy, she has lost her hair and needs a stem cell treatment, but she doesn't need a bone-marrow transplant.

And for that, she's lucky.

Hispanics needing bone marrow have a harder time finding matching donors than do other ethnicities because few Hispanics have registered to donate.

"That could've easily been me," Rodriguez said.

That's why Rodriguez, 31, is trying to raise awareness about the importance for Hispanics to give bone marrow. The former Zumba instructor is hosting a Zumba event Sunday afternoon that partly is a fundraiser for her ongoing cancer treatments and partly a campaign to encourage more people to become donors.

Among the 8 million people signed up as bone marrow donors, 800,000 or 10 percent, identify themselves as Hispanic or Latino. Though Hispanics comprise more than one-third of Texas' population, only 17 percent of registered bone marrow donors in the state are Hispanic.

Overall, Hispanics have a 72 percent chance of finding a bone marrow donor, compared with whites, who have a 93 percent chance, according to the donor program. Only blacks fare worse, with a 63 percent likelihood of finding a donor.

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Scientists turn skin cells into healthy heart tissue

By daniellenierenberg

Scientists turn skin cells into healthy heart tissue

Kate Kelland (Reuters) / 26 May 2012

The researchers said there were still many years of testing and refining ahead. But the results meant they might eventually be able to reprogramme patients cells to repair their own damaged hearts.

We have shown that its possible to take skin cells from an elderly patient with advanced heart failure and end up with his own beating cells in a laboratory dish that are healthy and young - the equivalent to the stage of his heart cells when he was just born, said Lior Gepstein, who led the work.

The researchers, whose study was published in the European Heart Journal on Wednesday, said clinical trials of the technique could begin within 10 years.

Heart failure is a debilitating condition in which the heart is unable to pump enough blood around the body. It has become more prevalent in recent decades as advances in medical science mean many more people survive heart attacks. At the moment, people with severe heart failure have to rely on mechanical devices or hope for a transplant.

Researchers have been studying stem cells from various sources for more than a decade, hoping to capitalise on their ability to transform into a wide variety of other kinds of cell to treat a range of health conditions.

There are two main forms of stem cells - embryonic stem cells, which are harvested from embryos, and reprogrammed human induced pluripotent stem cells (hiPSCs), often originally from skin or blood.

Gepsteins team took skin cells from two men with heart failure aged 51 and 61 and transformed them by adding three genes and then a small molecule called valproic acid to the cell nucleus.

They found that the resulting hiPSCs were able to differentiate to become heart muscle cells, or cardiomyocytes, just as effectively as hiPSCs that had been developed from healthy, young volunteers who acted as controls for the study.

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5 scientists receive stem-cell research grants

By daniellenierenberg

Five scientists from the University of California, San Diego and its School of Medicine have been awarded almost $12 million in new grants from the California Institute for Regenerative Medicine (CIRM) to conduct stem cell-based research into regenerating spinal cord injuries, repairing gene mutations that cause amyotrophic lateral sclerosis and finding new drugs to treat heart failure and Alzheimer's disease.

The awards mark the third round of funding in CIRM's Early Translational Awards program, which supports projects that are in the initial stages of identifying drugs or cell types that could become disease therapies. More than $69 million in awards were announced yesterday, including funding for first-ever collaboratively funded research projects with China and the federal government of Australia.

"With these new awards, the agency now has 52 projects in 33 diseases at varying stages of working toward clinical trials," said Jonathan Thomas, JD, PhD and CIRM governing board chair. "Californians should take pride in being at the center of this worldwide research leading toward new cures. These projects represent the best of California stem cell science and the best international experts who, together, will bring new therapies for patients."

The five new UC San Diego awards are:

CIRM was established in November 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Act. The statewide ballot measure provided $3 billion in funding for stem cell research at California universities and research institutions and called for the establishment of an entity to make grants and provide loans for stem cell research, research facilities, and other vital research opportunities.

The May 24 grants bring UC San Diego's total to more than $112 million in CIRM funding since the first awards in 2006.

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Israeli Scientists Reprogram Skin Cells into Beating Heart Tissue: Stem Cell Research Pays Off – Video

By raymumme

24-05-2012 09:53 For the first time ever, scientists have transformed normal skin cells into healthy beating heart tissue. Researchers based in Haifa in Israel, say they hope that the breakthrough will one day lead to new treatments for patients suffering from heart failure. Head of Research Professor Lior Gepstein "We were able to demonstrate the ability to take skin cells from very sick patients with significant heart failure, heart disease, and show that cells, skin cells from these patients can be eventually differentiated to become healthy heart cells in the dish. So one can take skin cells from a very sick individual, who has very sick heart cells, to reprogram them to become induced pluripotent stem cells and then make heart cells that are healthy, that are young and resemble heart cells at the day that the patient was born." At the moment, people with severe heart failure have to rely on mechanical devices or hope for a transplant. However, by studying stem cells from various sources for more than a decade, researchers are hoping to capitalise on their ability to transform stem cells into a wide variety of other kinds of cell. Head of Research Professor Lior Gepstein "These cells can be transplanted into hearts of animals, survive and function in synchrony with existing heart tissue. This study open the road, hopefully, to future clinical trials, in a decade or so, that will test the ability of such heart cells to repair the patient's own heart," There may be a lot to do before ...

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State awards stem cell grants to medical researchers

By JoanneRUSSELL25

SACRAMENTO California's stem cell agency today approved two grants to UC Davis Health System researchers for their innovative work in regenerative medicine.

Kyriacos A. Athanasiou, distinguished professor of orthopaedic surgery and professor and chair of biomedical engineering, and the Child Family Professor of Engineering at UC Davis, is investigating the use of skin-derived stem cells to heal cartilage injuries and debilitating conditions of the knee such as osteoarthritis.

W. Douglas Boyd, professor of surgery, plans to further refine a novel approach to treating cardiovascular injuries suffered during a heart attack by using stem cells and a tissue-like scaffold to repair cardiac damage.

The pair received individual grants totaling approximately $6.6 million from the California Institute for Regenerative Medicine's (CIRM) governing board.

Athanasiou's and Boyd's multi-year grants were among the proposals submitted to CIRM for its third round of Early Translational Awards, which are intended to enable clinical therapies to be developed more rapidly.

"Both of these scientists are conducting exciting research that could have far-reaching implications in health care," said Jan Nolta, director of the UC Davis Institute for Regenerative Cures and the university's stem cell program director. "Dr. Athanasiou is bioengineering new cartilage that could have the same physiological integrity as the cartilage a person is born with. Dr. Boyd is developing a treatment that uses a paper-thin patch embedded with stem cells to harness their regenerative powers to repair damaged heart muscle."

Boyd, who's a pioneering cardiothoracic surgeon, pointed out in his CIRM proposal that heart disease is the nation's number-one cause of death and disability. An estimated 16.3 million Americans over the age of 20 suffer from coronary heart disease, which in 2007 accounted for an estimated 1 in 6 deaths in the U.S. Boyd plans to use bone-marrow derived stem cells -- known as mesenchymal stem cells -- in combination with a bioengineered framework known as an extracellular matrix, to regenerate damaged heart tissue, block heart disease and restore cardiac function, something currently not possible except in cases of a complete and very invasive heart transplant.

An expert in biomedical engineering, Athanasiou is focusing on developing a cellular therapy using stem cells created from an individual's own skin -- known as autologous skin-derived stem cells -- which have shown great promise in animal models. He plans to use the new funding to conduct extensive toxicology and durability tests to determine the technique's long-term safety and efficacy. Such tests are among the many steps needed to advance toward human clinical trials.

Cartilage is the slippery tissue that covers the ends of bones in joints, allowing bones to glide over each other and absorbing the shock of movement. Cartilage defects from injuries and lifelong wear and tear can eventually degenerate into osteoarthritis. According to the National Institute of Arthritis and Musculoskeletal and Skin Diseases, osteoarthritis is the most common form of arthritis and affects an estimated 27 million Americans over the age of 25.

"For anyone suffering from osteoarthritis or other debilitating cartilage conditions, Dr. Athanasiou's goal of using stem cells to regenerate new tissue could have enormous quality-of-life and economic benefits," said Nolta, who is the recipient of a prior translational grant from CIRM to develop potential therapies for Huntington's disease . "Dr. Boyd's work is equally promising because he's using a bioengineered structure to encourage cardiac tissue repair, which could have important benefits in the treatment of heart disease."

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Recovery From Multiple Sclerosis By Growth Factor In Stem Cells

By JoanneRUSSELL25

Editor's Choice Main Category: Multiple Sclerosis Article Date: 24 May 2012 - 14:00 PDT

Current ratings for: 'Recovery From Multiple Sclerosis By Growth Factor In Stem Cells'

4.7 (10 votes)

4.5 (2 votes)

Animals that were injected with hepatocyte growth factor were noted to have grown new neural cells and lower levels of inflammation. Most significantly, the researchers noted that the protective envelope of myelin, the myelin sheath, which surrounds the core of a nerve fiber and facilitates the transmission of nerve impulses, re-grew and covered lesions that were caused by MS.

Robert H. Miller, professor of neurosciences at the School of Medicine and vice president for research at Case Western Reserve University declared: "The importance of this work is we think we've identified the driver of the recovery."

MS is caused by damage to the myelin sheath, the protective covering that surrounds nerve cells. The nerve damage is caused by inflammation, which occurs when the body's own immune cells attacks the nervous systems located in areas of the brain, the optic nerve, and spinal cord. This damage can cause an interruption of the nerve signals, which results in loss of balance and coordination, cognitive ability, as well as in other functions and in time, these intermittent losses may become permanent. In 2009, Caplan and Miller discovered that mice with MS injected with human mesenchymal stem cells recovered from the type of damage that was brought on by MS. A clinical trial is currently underway based on their research, whereby patients with MS are injected with their own stems cells.

During this trial, the team decided to first establish whether the presence of stem cells or other cells induce recovery. They injected a total of 11 animals with MS with the medium, in which mesenchymal stem cells that were taken from bone marrow grew, discovering that all animals displayed a rapid reduction in functional deficits. An analysis demonstrated that unless the injected molecules had a certain size or weight, i.e. between 50 and 100 kiloDaltons, the course of the disease remained unchanged.

Other research, as well as the team's own studies, suggested that this was likely to be instigated by the hepatocyte growth factor, which is secreted by mesenchymal stem cells.

The team then injected the animals with either 50 or 100 nanograms of the growth factor on alternate days for a 5-day period and observed a decrease in the level of signaling molecules that promote inflammation, whilst the level of signaling molecules that oppose inflammation increased. The researchers noted a growth of neural cells, whilst nerves that were exposed because of MS were rewrapped with myelin. Recovery was marginally better in those mice that received the 100-nanogram injections compared with those receiving the 50-nanogram injections.

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Skin Cells From Heart Failure Patients Made Into Healthy New Heart Muscle Cells

By Dr. Matthew Watson

Editor's Choice Main Category: Cardiovascular / Cardiology Article Date: 25 May 2012 - 0:00 PDT

Current ratings for: 'Skin Cells From Heart Failure Patients Made Into Healthy New Heart Muscle Cells'

4 (1 votes)

This achievement is significant, as it opens up the prospect of treating heart failure patients with their own, human-induced pluripotent stem cells (hiPSCs) to fix their damaged hearts.

Furthermore, the cells would avoid being rejected as foreign as they would be derived from the patients themselves. The study is published in the European Heart Journal. However, the researchers state that it could take a minimum of 5 to 10 years before clinical trials could start due to the many obstacles that must be overcome before using hiPSCs in humans is possible.

Although there has been advances in stem cell biology and tissue engineering, one of the major problems scientists have faced has been lack of good sources of human heart muscle cells and rejection by the immune system. Furthermore, until now, scientific have been unable to demonstrate that heart cells created from hiPSCs could integrate with existing cardiovascular tissue.

"What is new and exciting about our research is that we have shown that it's possible to take skin cells from an elderly patient with advanced heart failure and end up with his own beating cells in a laboratory dish that are health and young - the equivalent to the stage of his heart cells when he was just born," said Professor Lior Gepstein, Professor of Medicine (Cardiology) and Physiology at the Sohnis Research Laboratory for Cardiac Electrophysiology and Regenerative Medicine, Technion-Israel Institute of Technology and Rambam Medical Center in Haifa, Israel, who led the study.

In the study, Professor Gepstein, Ms Limor Zwi-Dantsis, and their colleagues retrieved skin cells from two male heart failure patients, aged 51 and 61 years, and reprogrammed the cells by delivering 3 transcription factors (Sox2, Oct4, and Klf4) in addition to a small molecule called valproic acid, to the cell nucleus. The team did not include a transcription factor called c-Myc as it is a known cancer-causing gene.

Professor Gepstein said:

In addition, the team used an alternative strategy involving a virus transferred reprogramming data to the cell nucleus. However, the team removed the virus after the information had been transferred in order to avoid insertional oncogenesis.

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Skin Cells From Heart Failure Patients Made Into Healthy New Heart Muscle Cells

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UC San Diego researchers receive new CIRM funding

By JoanneRUSSELL25

Public release date: 25-May-2012 [ | E-mail | Share ]

Contact: Scott LaFee slafee@ucsd.edu 619-543-6163 University of California - San Diego

Five scientists from the University of California, San Diego and its School of Medicine have been awarded almost $12 million in new grants from the California Institute for Regenerative Medicine (CIRM) to conduct stem cell-based research into regenerating spinal cord injuries, repairing gene mutations that cause amyotrophic lateral sclerosis and finding new drugs to treat heart failure and Alzheimer's disease.

The awards mark the third round of funding in CIRM's Early Translational Awards program, which supports projects that are in the initial stages of identifying drugs or cell types that could become disease therapies. More than $69 million in awards were announced yesterday, including funding for first-ever collaboratively funded research projects with China and the federal government of Australia.

"With these new awards, the agency now has 52 projects in 33 diseases at varying stages of working toward clinical trials," said Jonathan Thomas, JD, PhD and CIRM governing board chair. "Californians should take pride in being at the center of this worldwide research leading toward new cures. These projects represent the best of California stem cell science and the best international experts who, together, will bring new therapies for patients."

The five new UC San Diego awards are:

With a $1.8 million award, Lawrence Goldstein, PhD, professor in the Department of Cellular and Molecular Medicine, Howard Hughes Medical Institute Investigator and director of the UC San Diego Stem Cell Program, and colleagues will continue their work developing new methods to find and test drug candidates for Alzheimer's disease (AD). Currently, there is no effective treatment for AD. The researchers screen novel candidates using purified human brain cells made from human reprogrammed stem cells. Already, they have discovered that these human brain cells exhibit a unique biochemical behavior that indicates early development of AD in a dish.

Mark H. Tuszynski, MD, PhD, professor of neurosciences and director of the Center for Neural Repair at UC San Diego, and colleagues seek to develop more potent stem cell-based treatments for spinal cord injuries. By combining grafts of neural stem cells with scaffolds placed at injury sites, the researchers have reported substantial progress in restoring functional improvement in impaired animal models. The new $4.6 million grant will fund work to identify the optimal human neural stem cells for preclinical development and, in an unprecedented step, test this treatment in appropriate preclinical models of spinal cord injury, providing the strongest validation for human translation.

Amyotrophic lateral sclerosis or ALS (Lou Gehrig's disease) is a progressive neurological condition that is currently incurable. Gene Yeo, PhD, assistant professor in the Department of Cellular and Molecular Medicine, and colleagues will use a $1.6 million grant to exploit recent discoveries that specific mutations in RNA-binding proteins cause neuronal dysfunction and death. They will use neurons generated from patient cells containing the mutations to identify the unique RNA "signature" of these doomed neurons and screen for drug-like compounds that bypass the mutations to correct the RNA signature to obtain healthy neurons.

Eric David Adler, MD, an associate clinical professor of medicine and cardiologist, studies heart failure, including the use of stem cells to treat it. His $1.7 million award will fund research into Danon disease, a type of inherited heart failure that frequently kills patients by their 20s. Adler and colleagues will turn stem cells created from skin cells of patients with Danon disease into heart cells, then screen hundreds of thousands of drug candidates for beneficial effects. The most promising drugs will subsequently be tested on mice with a genetic defect similar to Danon disease, with the ultimate goal of identifying a suitable candidate for human clinical trials. The research may have broader applications for other conditions with similar pathogenesis, such as cancer and Parkinson's disease.

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UC San Diego researchers receive new CIRM funding

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UC San Diego Scientists Net $12 Million For Stem Cell Research

By daniellenierenberg

Five UC San Diego scientists have received almost $12 million combined from the California Institute for Regenerative Medicine to pay for stem cell-based research, the university announced today.

A team led by Lawrence Goldstein, of the Department of Cellular and Molecular Medicine and director of the UC San Diego Stem Cell Program, was given $1.8 million to continue looking for new methods to find and test possible medications for Alzheimer's disease, according to UCSD. They use reprogrammed stem cells in their work.

Dr. Mark Tuszynski, professor of neurosciences and director of the Center for Neural Repair, received $4.6 million to develop more potent stem cell-based treatments for spinal cord injuries.

Gene Yeo, assistant professor in the Department of Cellular and Molecular Medicine, was awarded $1.6 million to continue research into treatments for amyotrophic lateral sclerosis. His research hopes to take advantage of recent discoveries about ALS, or Lou Gehrig's disease, which center on mutations in RNA-binding proteins that cause dysfunction and death in neurons.

Dr. Eric David Adler, an associate clinical professor of medicine and cardiologist, was granted $1.7 million to screen potential drugs for Danon disease, a type of inherited heart failure that frequently kills patients by their 20s.

Yang Xu, a professor in the Division of Biological Sciences, was given $1.8 million to research the use of human embryonic stem cells to produce a renewable source of heart muscle cells that replace cells damaged or destroyed by disease, while overcoming biological resistance to new cells.

"With these new awards, the (institute) now has 52 projects in 33 diseases at varying stages of working toward clinical trials,'' said Jonathan Thomas, chairman of the CIRM governing board. "Californians should take pride in being at the center of this worldwide research leading toward new cures.''

CIRM was established in November 2004 with voter passage of the California Stem Cell Research and Cures Act. UC San Diego has received $112 million since CIRM began providing grants six years ago.

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Di'Anno Wants Former Iron Maiden Bandmate To Undergo Stem Cell Therapy

By daniellenierenberg

05/24/2012 . (Classic Rock) Former Iron Maiden singer Paul Di'Anno wants his ex-bandmate Clive Burr to undergo stem cell therapy, despite the costs and risks associated with the procedure.

Burr, the drummer with Maiden from 1979 until 1982, has been in a wheelchair as a result of multiple sclerosis, which has been attacking his nervous system since before he was diagnosed in 2002.

MS reduces the ability of the brain and spinal cord to communicate with each other, resulting in a wide range of potentially severe symptoms. The cause is unknown and there is no cure; but in 2009 researchers made the first breakthrough in reversing symptoms through stem cell therapy.

Di'Anno tells Talking Metal Pirate Radio Burr's condition is "not very good at all." He had a lot to say, read it here.

Classic Rock Magazine is an official news provider for antiMusic.com. Copyright Classic Rock Magazine- Excerpted here with permission.

antiMUSIC News featured on RockNews.info and Yahoo News

...end

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Di'Anno Wants Former Iron Maiden Bandmate To Undergo Stem Cell Therapy

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City of Hope Receives $5 Million Grant to Develop T Cell Treatment Targeting Brain Tumor Stem Cells

By LizaAVILA

DUARTE, Calif.--(BUSINESS WIRE)--

City of Hope was granted a $5,217,004 early translational research award by the California Institute for Regenerative Medicine (CIRM) to support the development of a T cell-based immunotherapy that re-directs a patients own immune response against glioma stem cells. City of Hope has been awarded more than $49.7 million in grant support from CIRM since awards were first announced in 2006.

City of Hope is a pioneer in T cell immunotherapy research, helping to develop genetically modified T cells as a treatment for cancer. This strategy, termed adoptive T cell therapy, focuses on redirecting a patients immune system to specifically target tumor cells, and has the potential to become a promising new approach for treatment of cancer.

In this research, we are genetically engineering a central memory T cell that targets proteins expressed by glioma stem cells, said Stephen J. Forman, M.D., Francis and Kathleen McNamara Distinguished Chair in Hematology and Hematopoietic Cell Transplantation and director of the T Cell Immunotherapy Research Laboratory. Central memory T cells have the potential to establish a persistent, lifelong immunity to help prevent brain tumors from recurring.

The American Cancer Society estimates that more than 22,000 people in the U.S. will be diagnosed with a brain tumor this year, and 13,700 will die from the disease. Glioma is a type of brain tumor that is often difficult to treat and is prone to recurrence. Currently, less than 20 percent of patients with malignant gliomas are living five years after their diagnosis. This poor prognosis is largely due to the persistence of tumor-initiating cancer stem cells, a population of malignant cells similar to normal stem cells in that they are able to reproduce themselves indefinitely. These glioma stem cells are highly resistant to chemotherapy and radiation treatments, making them capable of re-establishing new tumors.

Researchers at City of Hope previously have identified several proteins as potential prime targets for the development of cancer immunotherapies, such as interleukin 13 receptor alpha 2, a receptor found on the surface of glioma cells, and CD19, a protein that is active in lymphoma and leukemia cells. Both investigational therapies are currently in phase I clinical trials. Forman is the principal investigator for the newly granted study which will develop a T cell that targets different proteins expressed by glioma stem cells. Christine Brown, Ph.D., associate research professor, serves as co-principal investigator, and Michael Barish, Ph.D., chair of the Department of Neurosciences, and Behnam Badie, M.D., director of the Brain Tumor Program, serve as co-investigators on the project.

Because cancer stem cells are heterogeneous, our proposed therapy will target multiple antigens to cast as wide a net as possible over this malignant stem cell population, said Brown.

While in this effort, we are targeting a neurological cancer, our approach will lead to future studies targeting other cancers, including those that metastasize to the brain, added Barish.

The CIRM grant will help us to build a targeted T cell therapy against glioma that can offer lasting protection, determine the best way to deliver the treatment, establish an efficient process to manufacture these T cells for treatment, and get approval for a human clinical trial, said Badie.

City of Hope is also a collaborative partner providing process development, stem cell-derived cell products and regulatory affairs support in two other CIRM-funded projects that received early translational research grants. Larry Couture, Ph.D., senior vice president of City of Hopes Sylvia R. & Isador A. Deutch Center for Applied Technology Development and director of the Center for Biomedicine & Genetics, is working with Stanford University and Childrens Hospital of Orange County Research Institute on their respective projects.

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City of Hope Receives $5 Million Grant to Develop T Cell Treatment Targeting Brain Tumor Stem Cells

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Stem Cells Curb Chronic Pain in Mice

By NEVAGiles23

May 23, 2012 12:00pm

Replacing dead or dysfunctional nerve cells with new, healthy ones derived from stem cells eases chronic pain in mice, a new study found.

Researchers from the University of California, San Francisco coaxed mouse embryonic stem cells into becoming mature nerve cells that could bridge gaps in the circuitry that triggers neuropathic pain.

One of the major causes of neuropathic pain is the loss of inhibitory control at the level of the spinal cord because of nerve loss or dysfunction, said study author Allan Basbaum, chairman of UCSFs department of anatomy. The idea was to replace or repopulate the spinal cord cells that provide that inhibition.

The same stem cells, destined to become inhibitory neurons that dampen the signals that cause pain, were previously shown to improve symptoms in a mouse model of epilepsy, Basbaum said. The question was whether we could take the exact same cells and put them in the spinal cord.

Before injecting the cells into the spinal cords of mice with neuropathic pain, the researchers labeled them with a fluorescent tracer to track the connections they made.

We were able to show how these cells integrate beautifully, Basbaum said, describingthe waythe transplanted cells looked and behaved like the mouses own.

Not only did the cells set up shop in the spinal cord, sending and receiving signals through a complex network of neurons, they also eased the neuropathic pain.

In four weeks, the animals condition completely disappeared, Basbaum said, adding that transplanted control cells that lacked the inhibitory properties of the stem-cell-derived neurons failed to ease the pain.

The clinical significance is that we think were actually modifying the disease, not just treating the symptoms, Basbaum said, adding that drugs currently used to ease neuropathic pain fail to treat the underlying problem. Instead of taking a drug to suppress the pain, were trying to normalize the circuit that was damaged by the disease or the injury. The cells repopulate, they integrate, and basically they treat the disease.

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How damaged hearts could be healed by growing stem cells

By LizaAVILA

By Jenny Hope

PUBLISHED: 18:09 EST, 22 May 2012 | UPDATED: 01:35 EST, 23 May 2012

Scientists claim they can rejuvenate broken hearts using skin cells that have been turned into heart muscle cells.

New research opens up the prospect of reprogramming cells taken from heart failure patients that would not be rejected by their bodies.

It is the first time that stem cells taken from the skin of elderly and diseased patients - who are most likely to need such treatment - have been transformed into heart cells.

New developments: The research opens up the prospect of reprogramming cells taken from heart failure patients that would not be rejected by their bodies

Previously skin cells taken from young and healthy people have been transformed into heart muscle cells.

But researchers from Israel warn that clinical trials could be a decade away, as more work in the laboratory and major investment are needed.

The research is the latest advance in stem cell therapy where the intention is to infused repair cells directly into the scarred heart muscle of patients suffering debilitating symptoms such as breathlessness and fatigue.

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How damaged hearts could be healed by growing stem cells

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Could Stem Cells Cure MS?

By JoanneRUSSELL25

A growth factor isolated from human stem cells shows promising results in a mouse model of multiple sclerosis.

Human mesenchymal stem cells (hMSCs) have become a popular potential therapy for numerous autoimmune and neurological disorders. But while these bone marrow-derived stem cells have been studied in great detail in the dish, scientists know little about how they modulate the immune system and promote tissue repair in living organisms.

Now, one research team has uncovered a molecular mechanism by which hMSCs promote recovery in a mouse model of multiple sclerosis (MS).

According to research, published online Sunday (May 20) in Nature Neuroscience, a growth factor produced by hMSCs fights MS in two ways: blocking a destructive autoimmune response and repairing neuronal damage. The finding could help advance ongoing clinical trials testing hMSCs as a therapy for MS.

The researchers have identified a unique factor that has surprisingly potent activity mediating neuron repair, said Jacques Galipeau, a cell therapy researcher at Emory University in Atlanta, Georgia, who was not involved in the research. The magnitude of the effect on a mouse model of MS is a big deal.

MS is an autoimmune disease in which the immune system attacks myelin sheaths that surround and protect nerve cells. The attack leaves nerves exposed and unable to send signals to the brain and back, resulting in the loss of motor skills, coordination, vision, and cognitive abilities. There is no cure for MS, and most current therapies work to simply suppress the immune system, preventing further neuronal damage. None have demonstrated an ability to also repair damaged myelin and promote recovery.

In 2009, Robert Miller and colleagues at Case Western Reserve University in Cleveland, Ohio, demonstrated that hMSCs dramatically reversed the symptoms of multiple sclerosis in a mouse model of the disorder. The animals got better, recalled Miller. The team hypothesized that the stem cells suppress the immune response and promote remyelination.

But Miller wanted to know exactly what the cells were doing. To find out, his team isolated the medium on which the hMSCs were grown to determine if the cells or something they secreted was responsible for the observed recovery. The medium alone was enough to induce recovery in mice, pointing to the latter.

To find out exactly which molecule or molecules in the medium were responsible, the researchers separated the proteins in the fluid based on the molecular weight and injected each isolate into mice exhibiting symptoms of MS. The mid-weight solution, of proteins with masses between 50 and 100 kilodaltons (kDa), caused recovery. That eliminated a huge number of potential candidates, said Miller.

The researchers then narrowed the field again with a literature search for a molecule that fit their criteria: secreted by hMSCs, 50-100 kDa in size, and involved in tissue repair. They identified hepatocyte growth factor (HGF), a cytokine made by mesenchymal cells that has been shown to promote tissue regeneration and cell survival in numerous experiments. Sure enough, HGF alone was enough to promote recovery in the MS mouse models, and blocking the receptor for HGF in those mice blocked recovery. The team also demonstrated that HGF suppresses immune responses in vivo and accelerates remyelination of neurons in vitro. Finally, they saw that HGF causes remyelination in rats with a lesion on their spinal cord.

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